JP2015507478A - Ｃａｓｐ２に対する二本鎖ｒｎａ化合物およびその使用 - Google Patents

Ｃａｓｐ２に対する二本鎖ｒｎａ化合物およびその使用 Download PDF

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Publication number
JP2015507478A
JP2015507478A JP2014551296A JP2014551296A JP2015507478A JP 2015507478 A JP2015507478 A JP 2015507478A JP 2014551296 A JP2014551296 A JP 2014551296A JP 2014551296 A JP2014551296 A JP 2014551296A JP 2015507478 A JP2015507478 A JP 2015507478A
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compound
eye
use according
glaucoma
injury
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JP2015507478A5 (enExample
Inventor
シャイ エルリッヒ
シャイ エルリッヒ
ジェイムズ ディー． トンプソン
ジェイムズ ディー． トンプソン
ラビア オズデン
ラビア オズデン
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Quark Pharmaceuticals Inc
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Quark Pharmaceuticals Inc
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/08—Solutions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M5/00—Devices for bringing media into the body in a subcutaneous, intra-vascular or intramuscular way; Accessories therefor, e.g. filling or cleaning devices, arm-rests
    • A61M5/002—Packages specially adapted therefor, e.g. for syringes or needles, kits for diabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/04—Artificial tears; Irrigation solutions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1135—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against oncogenes or tumor suppressor genes
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    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/22—Cysteine endopeptidases (3.4.22)
    • C12Y304/22055—Caspase-2 (3.4.22.55)
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

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JP2014551296A 2012-01-04 2013-01-03 Ｃａｓｐ２に対する二本鎖ｒｎａ化合物およびその使用 Withdrawn JP2015507478A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201261582886P 2012-01-04 2012-01-04
US61/582,886 2012-01-04
US201261596231P 2012-02-08 2012-02-08
US61/596,231 2012-02-08
PCT/US2013/020012 WO2013103632A1 (en) 2012-01-04 2013-01-03 Double-stranded rna compounds to casp2 and uses thereof

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US (2) US9382542B2 (enExample)
EP (1) EP2800812A1 (enExample)
JP (2) JP2015507478A (enExample)
KR (2) KR20140109430A (enExample)
CN (1) CN104053776A (enExample)
AU (1) AU2013206951B2 (enExample)
BR (1) BR112014016640A2 (enExample)
CA (1) CA2858627C (enExample)
EA (1) EA036003B1 (enExample)
IL (1) IL233142B (enExample)
NZ (1) NZ625980A (enExample)
WO (1) WO2013103632A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015136255A1 (en) * 2014-03-11 2015-09-17 The University Of Birmingham Combined inhibition of caspase-2 and caspase-6 in neuroprotection and axon regeneration
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic agents

Citations (2)

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WO2010048352A2 (en) * 2008-10-22 2010-04-29 Quark Pharmaceuticals, Inc. Methods for treating eye disorders
WO2010117077A1 (ja) * 2009-04-10 2010-10-14 参天製薬株式会社 シロリムス誘導体を有効成分として含有する網脈絡膜疾患の治療剤

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US6444676B1 (en) * 1999-12-20 2002-09-03 Iok-Hou Pang Use of PARP inhibitors in the treatment of glaucoma
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JP2010507387A (ja) * 2006-10-25 2010-03-11 クアーク・ファーマスーティカルス、インコーポレイテッド 新規のｓｉＲＮＡおよびその使用方法
WO2008094860A2 (en) * 2007-01-30 2008-08-07 Allergan, Inc. Treating ocular diseases using peroxisome proliferator-activated receptor delta antagonists
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US20120135009A1 (en) * 2009-07-16 2012-05-31 Pascale Giraudon Cleaved and Phosphorylated CRMP2 as Blood Marker of Inflammatory Diseases of the Central Nervous System
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Publication number Priority date Publication date Assignee Title
WO2010048352A2 (en) * 2008-10-22 2010-04-29 Quark Pharmaceuticals, Inc. Methods for treating eye disorders
WO2010117077A1 (ja) * 2009-04-10 2010-10-14 参天製薬株式会社 シロリムス誘導体を有効成分として含有する網脈絡膜疾患の治療剤

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CN104053776A (zh) 2014-09-17
US20140371439A1 (en) 2014-12-18
AU2013206951B2 (en) 2016-11-24
BR112014016640A2 (pt) 2021-03-16
CA2858627C (en) 2020-12-01
KR102202004B1 (ko) 2021-01-12
US9382542B2 (en) 2016-07-05
AU2013206951A1 (en) 2014-07-03
WO2013103632A1 (en) 2013-07-11
JP6774928B2 (ja) 2020-10-28
KR20190137940A (ko) 2019-12-11
CA2858627A1 (en) 2013-07-11
EP2800812A1 (en) 2014-11-12
EA036003B1 (ru) 2020-09-11
US20160304879A1 (en) 2016-10-20
JP2018099117A (ja) 2018-06-28
IL233142A0 (en) 2014-07-31
KR20140109430A (ko) 2014-09-15
IL233142B (en) 2019-01-31
EA201491048A1 (ru) 2015-09-30
NZ625980A (en) 2015-05-29
US10570394B2 (en) 2020-02-25

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